SLDB — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-12

• On March 11, 2026, we announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical trial as of a February 23, 2026 data cutoff date. For a full description of the initial results from the INSPIRE DUCHENNE trial, see Part I, Item I, “Business” in our Annual Report on Form 10-K fil…

• SGT-003 has been generally well tolerated in the 47 participants dosed in the Phase 1/2 INSPIRE DUCHENNE clinical trial as of May 11, 2026. The safety and tolerability profile observed in the INSPIRE DUCHENNE trial continued to be promising; SGT-003 is administered using a low-burden, steroid-only p…

• Enrollment and dosing in the INSPIRE DUCHENNE trial is ongoing and being conducted at 15 clinical sites across the United States, Canada, Italy and the United Kingdom. We believe we have aligned with the FDA on SGT-003's potency assay strategy and will continue commercial-readiness CMC activities, w…

• In October 2025, we activated the first clinical trial site and began screening participants for IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled trial evaluating SGT-003. In February 2026, we announced positive feedback from a Type C meeting with the FDA where we reached alig…

• We expect to continue discussions with the FDA as we seek guidance on a potential accelerated approval pathway for SGT-003 and we expect to provide an update as regulatory discussions progress.

Text removed vs the prior filing · source: 10-Q · 2025-11-03

• On November 3, 2025, we announced positive interim data from the Phase 1/2 INSPIRE DUCHENNE trial as of the data cutoff date of September 29, 2025. Interim biopsy data reported from 10 participants treated with SGT-003 showed a mean microdystrophin expression of 58%, as measured by both western blot…

• As of October 31, 2025, 23 participants have been dosed in the INSPIRE DUCHENNE trial and SGT-003 has been generally well tolerated as of such date. There has been one treatment-related serious adverse event reported in the INSPIRE DUCHENNE trial. This serious adverse event was identified as a Grade…

• For a more detailed description of the interim results from the INSPIRE DUCHENNE trial, see our Form 8-K filed November 3, 2025.

• Enrollment and dosing in the INSPIRE DUCHENNE trial is ongoing, with 30 total participants anticipated to be dosed by early 2026.

• The INSPIRE DUCHENNE trial currently has 15 active sites across the U.S., Canada, Italy and the United Kingdom, with an additional site expected to be activated by the end of 2025. A meeting with the FDA to discuss potential regulatory pathways, including accelerated approval pathways, for SGT-003 i…

Text added vs the prior filing · source: 10-Q · 2026-05-12

• Our management, with the participation of our President and Chief Executive Officer and our Chief Financial Officer (our principal executive officer and principal financial and accounting officer, respectively), evaluated the effectiveness of our disclosure controls and procedures as of March 31, 20…

• under the Securities Exchange Act of 1934, as amended (the “Exchange Act”), means controls and other procedures of a company that are designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is recorded, processed, summa…

• In designing and evaluating our disclosure controls and procedures, management recognizes that disclosure controls and procedures, no matter how well designed and operated, can provide only reasonable, not absolute, assurance that the objectives of the disclosure controls and procedures are met. Add…

• There have been no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) that have occurred during the three months ended March 31, 2026 that have materially affected, or are reasonably likely to materially affect, our internal …

Text removed vs the prior filing · source: 10-Q · 2025-11-03

• Our management, with the participation of our President and Chief Executive Officer and our Chief Financial Officer (our principal executive officer and principal financial and accounting officer, respectively), evaluated the effectiveness of our disclosure controls and procedures as of September 30…

• In designing and evaluating our disclosure controls and procedures, management recognizes that disclosure controls and procedures, no matter how well designed and operated, can provide only reasonable, not absolute, assurance that the objectives of the disclosure controls and procedures are met. Add…

• There have been no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) that have occurred during the three months ended September 30, 2025 that have materially affected, or are reasonably likely to materially affect, our inter…

Text added vs the prior filing · source: 10-Q · 2026-05-12

• There have been several significant adverse side effects in gene therapy treatments in the past, including reported cases of leukemia and death seen in other clinical trials. The FDA convened the Cellular, Tissue, and Gene Therapies Advisory Committee in September 2021 to discuss toxicity risks of A…

• toxicity). While new recombinant capsids have been developed with the intent to reduce these side effects, gene therapy is still a relatively new approach to disease treatment and additional adverse side effects could develop. There have been reports of significant adverse side effects, including mu…

• Adverse side effects may be observed following administration of any AAV gene therapy, including SGT-003 or other Candidates. Not all contemplated AAV delivery systems have been validated in human clinical trials previously, such as POLARIS-101TM, which is a novel capsid. If a delivery system does n…

• Additionally, if one or more of our Candidates receive marketing approval, the FDA could require us to adopt a Risk Evaluation and Mitigation Strategy (“REMS”) to ensure that the benefits outweigh the risks, which may include, among other things, a medication guide outlining the risks of the product…

• all. A failure of one or more clinical trials can occur at any stage of testing. Events that may prevent successful or timely completion of clinical development include:

Text removed vs the prior filing · source: 10-Q · 2025-11-03

• advance our other Candidates into clinical trials, including our planned ARTEMIS trial of SGT-501;

• There have been several significant adverse side effects in gene therapy treatments in the past, including reported cases of leukemia and death seen in other clinical trials. The FDA convened the Cellular, Tissue, and Gene Therapies Advisory Committee in September 2021 to discuss toxicity risks of A…

• Adverse side effects may be observed following administration of any AAV gene therapy, including SGT-003 or other Candidates. Not all contemplated AAV delivery systems have been validated in human clinical trials previously, such as AAV‑SLB101, which is a novel capsid. If a delivery system does not …

• Additionally, if one or more of our Candidates receive marketing approval, the FDA could require us to adopt a Risk Evaluation and Mitigation Strategy ("REMS") to ensure that the benefits outweigh the risks, which may include, among other things, a medication guide outlining the risks of the product…

• the final data are available. Material adverse changes in the final data compared to preliminary or interim data could significantly harm our business prospects.